CAMBRIDGE, Mass.--(BUSINESS WIRE)--May. 2, 2012--
AVEO Pharmaceuticals Inc. (NASDAQ: AVEO) announced today preliminary
data from the exploratory, randomized Phase 2 study comparing the
combination of ficlatuzumab and gefitinib, an EGFR tyrosine kinase
inhibitor (TKI), to gefitinib monotherapy in previously untreated Asian
subjects with non-small cell lung cancer (NSCLC), a population with a
high prevalence of EGFR sensitizing mutations (SM+). In this study,
encouraging signals of activity were observed in unique subsets of
patients based on EGFR mutation status and c-Met expression level. EGFR
TKI therapies are the standard of care for patients with EGFR SM+.

“Some patients with EGFR sensitizing mutations do not respond well to
EGFR TKI therapy,” stated Tony Mok, M.D., professor, Department of
Clinical Oncology, The Chinese University of Hong Kong, and senior
investigator of the Phase 2 trial. “This study has uncovered an
important subset of patients with non-small cell lung cancer that may be
underserved by EGFR TKIs. Patients with EGFR sensitizing mutations and
low c-Met expression levels treated with the combination of ficlatuzumab
and gefitinib lived twice as long without their disease progressing
compared to those treated with gefitinib alone. These data support a
potential benefit from combining an EGFR targeted therapy with
ficlatuzumab and warrant further investigation in lung cancer.”

The primary endpoint of the study was overall response rate (ORR), and
progression free survival (PFS) was a secondary endpoint. Preliminary
results in the intent-to-treat (ITT) population (n=94 each arm) showed a
trend favoring the ficlatuzumab/gefitinib combination; however, as with
data from Phase 2 studies in NSCLC with other inhibitors of the Met
pathway, study results in the ITT population did not reach statistical
significance. In the ITT population, ORR was 43% for the
ficlatuzumab/gefitinib combination armversus 40% for the
gefitinib monotherapy arm, and median PFS was 5.6 months versus 4.7
months for the ficlatuzumab/gefitinib combination arm versus the
gefitinib monotherapy arm, respectively, favoring the combination arm.

Encouraging signals of activity were observed in biomarker subset
populations. Key findings are summarized in the following chart:

Biomarker Subset

Ficlatuzumab/Gefitinib Combination

Gefitinib Alone

# ofSubjects

Median PFS(months)

ORR,%

# ofSubjects

Median PFS(months)

ORR,%

EGFR SM+/c-Met Low

10

11.0

70

9

5.5

44

EGFR SM+/c-Met High

23

9.2

52

27

9.2

63

EGFR SM-/c-Met Low

9

1.3

0

13

2.3

0

EGFR SM-/c-Met High

11

1.8

27

16

1.8

13

No clinically meaningful differences in adverse event rates between the
combination and monotherapy arms were observed, and the combination was
well-tolerated. Complete data from this trial, which will include
biomarker analyses, are anticipated to be submitted for presentation at
an upcoming medical meeting in the second half of this year.

“These are important data for driving our ficlatuzumab program forward,
as well as for demonstrating the clinical value of AVEO’s proprietary
Human Response Platform,” stated William Slichenmyer, M.D., Sc.M., chief
medical officer of AVEO. “Through our platform, we were able to
recognize the interplay between the EGFR and HGF/c-Met pathways. These
biomarker insights, combined with the clinical data, support our
continued development of ficlatuzumab in combination with an EGFR
inhibitor. We look forward to further clinical investigation in lung
cancer. In addition, we will be initiating a clinical study in head and
neck cancer later this year.”

Study Overview

The open-label, two-arm, randomized exploratory Phase 2 study was
designed to compare the combination of ficlatuzumab and gefitinib versus
gefitinib monotherapy, in clinically selected Asian subjects with
previously untreated advanced NSCLC who have a high likelihood of
harboring activating EGFR mutations. Ninety-four(94) patients
were randomized to gefitinib and ficlatuzumab/gefitinib arms,
respectively; 144 tumor tissue samples were available for biomarker
analysis. Subjects who demonstrated disease control (complete response,
partial response, or stable disease for 12 weeks or longer) in the
gefitinib alone arm were eligible to cross-over upon progression to a
combination of gefitinib and ficlatuzumab to assess whether acquired
resistance to gefitinib can be overcome with the addition of
ficlatuzumab.

About Ficlatuzumab and the HGF/c-MET Pathway

HGF is a ligand that binds to and activates a receptor called c-Met.
Activation of the HGF/c-Met pathway is believed to be important in
normal processes in embryonic development and wound healing, but is also
believed to trigger many activities involved in cancer development and
metastasis. HGF/c-Met has been shown to be one of the most potent
drivers of tumor growth in AVEO’s Human Response Platform.

HGF/c-Met over-expression is observed in many solid tumors including
breast, colorectal, gastric, head and neck, lung and prostate, as well
as hematologic malignancies1. Additionally, c-Met and EGFR
are frequently co-amplified and co-expressed in a variety of tumor
types; HGF/c-Met pathway upregulation can render EGFR-targeted therapy
resistance, and vice-versa2-4.

Ficlatuzumab is a humanized IgG1 antibody that binds to the HGF ligand
with high affinity and specificity to inhibit the biological activities
of the HGF/c-Met pathway.

About AVEO

AVEO Pharmaceuticals (NASDAQ: AVEO) is a cancer therapeutics company
committed to discovering, developing and commercializing targeted
therapies to impact patients’ lives. AVEO’s proprietary Human Response
Platform™ provides the company unique insights into cancer biology and
is being leveraged in the discovery and clinical development of its
cancer therapeutics. For more information, please visit the company’s
website at www.aveopharma.com.

Cautionary Note Regarding Forward-Looking Statements

Any statements in this press release about AVEO’s future
expectations, plans and prospects, including statements about: the
potential efficacy and safety of ficlatuzumab; advancement of the
ficlatuzumab clinical development plans in lung cancer and head and neck
cancer; ficlatuzumab’s therapeutic potential in combination with EGFR
targeted therapies; the potential of AVEO’s cancer biology platform and
biomarker capabilities to offer a unique advantage in oncology drug
development; and other statements containing the words "believes,"
"anticipates," "plans," "expects," "potential," "will" and similar
expressions, constitute forward-looking statements within the meaning of
The Private Securities Litigation Reform Act of 1995. Actual results may
differ materially from those indicated by such forward-looking
statements as a result of various important factors, including risks
relating to: AVEO’s ability to successfully research, develop and obtain
and maintain regulatory approvals for ficlatuzumab and its other product
candidates, including risks relating to its ability to successfully
advance clinical development of ficlatuzumab for the treatment of lung
cancer; the possibility that favorable historical preclinical and
clinicaltrial results may not be predictive of the results in
future preclinical and clinical trials; AVEO’s ability to obtain and
maintain adequate protection for intellectual property rights relating
to its product candidates and technologies; unplanned operating
expenses; AVEO’s ability to raise substantial additional funds to
achieve its goals, including with respect to the further development of
ficlatuzumab; competition; general economic and industry conditions; and
other factors discussed in the "Risk Factors" section of AVEO’s Annual
Report on Form 10-K filed with the Securities and Exchange Commission,
and in other filings that AVEO periodically makes with the SEC. In
addition, the forward-looking statements included in this press release
represent AVEO’s views as of the date of this press release. AVEO
anticipates that subsequent events and developments will cause its views
to change. However, while AVEO may elect to update these forward-looking
statements at some point in the future, AVEO specifically disclaims any
obligation to do so. These forward-looking statements should not be
relied upon as representing AVEO’s views as of any date subsequent to
the date of this press release.